A Study of BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation (ATLAS)

UK Chief Investigator

Professor Dame Pamela Shaw

Research summary

Amyotrophic lateral sclerosis (ALS), is a rapidly progressive and fatal neurological disease that attacks the nerve cells (neurons) that control voluntary muscles such as those in the arms, legs, and face.

Although the majority of patients suffer from sporadic ALS without family history, approximately 2%, have an inherited form of ALS caused by a variety of genetic mutations known as superoxide dismutase 1 mutations (SOD1-ALS).

This study is being carried out to find out more about the study drug, BIIB067 as a potential treatment for people with SOD1 ALS. The optimum timing for starting BIIB067 in this group of people is currently unknown (i.e., prior to or after the appearance of clinically manifested disease). This study will involve people who have 1 of a limited number of possible SOD1 gene mutations and who are not showing any signs or symptoms of ALS.

The study consists of 4 parts. In Part A (run-in period) the participant’s neurofilament (NF) level will be monitored. NF is a protein with many functions in the body. This study will use NF as a biomarker (naturally occurring substances in the body that change as a result of disease). No study treatment will be given. If NF levels increase and the participant is not showing signs or symptoms of ALS, the participant will be screened to enter Part B (randomised placebo-controlled in pre-symptomatic participants with elevated NfL). If the participant shows signs or symptoms of ALS during Part B, they will be screened and entered into Part C of the study (open-label extension phase). If the participant shows signs or symptoms of ALS during Part A of the study or during screening for Part B of the study, they may be eligible to enter Part D of the study (randomised placebo-controlled in participants with clinically manifested ALS).

Around 150 participants will be enrolled in Part A, of which around 28 will be enrolled in Part B/C and approximately 6 will be enrolled in Part D. Approximately 30 sites are planned globally. 

Current status

Open to recruitment

Recruitment target

4 participants in the UK; up to 150 participants globally

Recruitment group(s)

Pre-symptomatic participants with SOD1 mutations


Information about study sites

Contact details


Key dates

Recruitment open date: May 2022

Planned recruitment end date: 17 September 2022

Inclusion / exclusion criteria

Key Part A Inclusion criteria

Key Part A Exclusion criteria





Study design

Interventional; multi-arm, multi-stage, adaptive, randomised controlled trial




Outcome measures

Primary outcome measure

Secondary outcome measures